Patient perspective missing in device clinical trials, FDA says
- FDA's Patient Engagement Advisory Committee is seeking public comment to inform its draft guidance on developing patient-oriented medical device clinical trials. The agency's device unit considers patient engagement "activities that involve patient stakeholders sharing their experiences, perspectives, needs, and priorities to help inform the design, the implementation, and dissemination for medical product development and assessment."
- Clinical trials for devices have historically struggled to recruit, retain and support diverse, independent participants, leaving FDA with limited evidence when approving devices.
- The call for feedback follows an October 2017 discussion by the panel, the first of two inaugural meetings of the group chartered in 2017 to more meaningfully include patients in the device regulation process. It also comes in a week when more consumers are gaining awareness of how FDA approves devices after a medical device safety investigation rolled out from the International Consortium of Investigative Journalists, NBC News and others.
Under the existing 510(k) approval pathway, which FDA proposed stringent updates to Monday, most medical devices that prove substantial similarity to an already approved product do not have to run clinical trials prior to gaining premarket approval.
When clinical trials for devices are sponsored, nearly half of those trials do not meet target enrollment, according to FDA. Additionally, changes to trial protocols over the course of a study often cause longer and costlier investigations, leading to poor participant retention and "increased unnecessary burden and risk exposure" to patients, FDA said.
Critics say that insufficient clinical trials can lead to overly hasty PMAs, and the fact that FDA can be reluctant to pull devices off the market further emphasizes the importance of availability of thorough evidence prior to approval.
"How to get better treatment options available quickly should be discussed in the context of how to protect patients from harm. Those two things should be looked at at the same time. But that's not usually done," said Diana Zuckerman, president of the National Center for Health Research, who participated in PEAC's meetings.
"The concern at numerous patient meetings at the FDA has been that too many of the people participating are often recruited by industry to lobby for faster approvals and lower standards rather than to talk about patient safety, and yet we know that patients care about patient safety and that they don't want to be harmed by devices that have risks that they were never told about," she said.
The forthcoming FDA guidance aims to "demystify" the nature and purpose of patient engagement in the clinical trial process.
"By engaging patients throughout the process, clinical trials may become more patient-centric and reflect patient values, leading to improved clinical trial quality and greater uptake of results by patients and providers when making treatment decisions, ultimately leading to earlier U.S. patient access to beneficial medical devices," the committee wrote.
So far, the patient committee's potential strategies for empowered patient engagement include increasing opportunities for remote data collection using mobile technologies, to better convenience long-range trial participants and allow for submission of patient reported outcomes, which better reflect outcomes important to patients. The panel also proposed collaborating with patient advocacy groups to better communicate trial outcomes to patients.
The nine-member committee was first announced in September 2015 and the official charter for the committee was filed Oct. 6, 2017. The panel was sanctioned to explore such topics as device-related quality of life or health status issues, patient preference study design, and patient reported outcomes.
The comment period will continue through Jan. 8, 2019.
- FDA Patient Engagement Advisory Committee Patient Engagement in Medical Device Clinical Trials